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974 results

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Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.
Harmatz P, Yu ZF, Giugliani R, Schwartz IV, Guffon N, Teles EL, Miranda MC, Wraith JE, Beck M, Arash L, Scarpa M, Ketteridge D, Hopwood JJ, Plecko B, Steiner R, Whitley CB, Kaplan P, Swiedler SJ, Hardy K, Berger KI, Decker C. Harmatz P, et al. Among authors: hardy k. J Inherit Metab Dis. 2010 Feb;33(1):51-60. doi: 10.1007/s10545-009-9007-8. Epub 2010 Feb 6. J Inherit Metab Dis. 2010. PMID: 20140523 Free PMC article. Clinical Trial.
Respiratory and sleep disorders in mucopolysaccharidosis.
Berger KI, Fagondes SC, Giugliani R, Hardy KA, Lee KS, McArdle C, Scarpa M, Tobin MJ, Ward SA, Rapoport DM. Berger KI, et al. J Inherit Metab Dis. 2013 Mar;36(2):201-10. doi: 10.1007/s10545-012-9555-1. Epub 2012 Nov 15. J Inherit Metab Dis. 2013. PMID: 23151682 Free PMC article. Review.
Long term effects of denufosol tetrasodium in patients with cystic fibrosis.
Ratjen F, Durham T, Navratil T, Schaberg A, Accurso FJ, Wainwright C, Barnes M, Moss RB; TIGER-2 Study Investigator Group. Ratjen F, et al. J Cyst Fibros. 2012 Dec;11(6):539-49. doi: 10.1016/j.jcf.2012.05.003. Epub 2012 Jun 8. J Cyst Fibros. 2012. PMID: 22682898 Free article. Clinical Trial.
Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease.
Walters MC, Hardy K, Edwards S, Adamkiewicz T, Barkovich J, Bernaudin F, Buchanan GR, Bunin N, Dickerhoff R, Giller R, Haut PR, Horan J, Hsu LL, Kamani N, Levine JE, Margolis D, Ohene-Frempong K, Patience M, Redding-Lallinger R, Roberts IA, Rogers ZR, Sanders JE, Scott JP, Sullivan KM; Multicenter Study of Bone Marrow Transplantation for Sickle Cell Disease. Walters MC, et al. Among authors: hardy k. Biol Blood Marrow Transplant. 2010 Feb;16(2):263-72. doi: 10.1016/j.bbmt.2009.10.005. Epub 2009 Oct 12. Biol Blood Marrow Transplant. 2010. PMID: 19822218 Free PMC article.
Asthma in sickle cell disease.
Newaskar M, Hardy KA, Morris CR. Newaskar M, et al. ScientificWorldJournal. 2011 May 26;11:1138-52. doi: 10.1100/tsw.2011.105. ScientificWorldJournal. 2011. PMID: 21623460 Free PMC article. Review.
Probability of treatment following acute decline in lung function in children with cystic fibrosis is related to baseline pulmonary function.
Morgan WJ, Wagener JS, Yegin A, Pasta DJ, Millar SJ, Konstan MW; Scientific Advisory Group, investigators; coordinators of the Epidemiologic Study of Cystic Fibrosis. Morgan WJ, et al. J Pediatr. 2013 Oct;163(4):1152-7.e2. doi: 10.1016/j.jpeds.2013.05.013. Epub 2013 Jun 27. J Pediatr. 2013. PMID: 23810128 Free PMC article.
Novel CFTR variants identified during the first 3 years of cystic fibrosis newborn screening in California.
Prach L, Koepke R, Kharrazi M, Keiles S, Salinas DB, Reyes MC, Pian M, Opsimos H, Otsuka KN, Hardy KA, Milla CE, Zirbes JM, Chipps B, O'Bra S, Saeed MM, Sudhakar R, Lehto S, Nielson D, Shay GF, Seastrand M, Jhawar S, Nickerson B, Landon C, Thompson A, Nussbaum E, Chin T, Wojtczak H; California Cystic Fibrosis Newborn Screening Consortium. Prach L, et al. J Mol Diagn. 2013 Sep;15(5):710-22. doi: 10.1016/j.jmoldx.2013.05.006. Epub 2013 Jun 28. J Mol Diagn. 2013. PMID: 23810505 Free PMC article.
974 results